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| Name | Class |
|---|---|
| Hoffmann-La Roche | INDUSTRY |
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Cohort 8 (non-ambulatory participants) is currently enrolling new participants. Enrollment for Cohorts 1 through 7 has been completed.
This is an open-label gene transfer therapy study evaluating the safety of and expression from delandistrogene moxeparvovec in participants with Duchenne Muscular Dystrophy (DMD). The maximum participant duration for this study is 156 weeks.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Delandistrogene Moxeparvovec | Experimental | Participants will receive a single intravenous (IV) infusion of delandistrogene moxeparvovec on Day 1. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| delandistrogene moxeparvovec | Genetic | Single IV infusion of delandistrogene moxeparvovec |
|
| Measure | Description | Time Frame |
|---|---|---|
| Part 1 (Cohorts 1 to 5): Change from Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12, as Measured by Western Blot | Baseline, Week 12 | |
| Part 1 (Cohorts 6 to 8): Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12 as Measured by Western Blot | Week 12 | |
| Cohort 8: Number of Participants with Acute Liver Injury (ALI) | Baseline up to Week 72 |
| Measure | Description | Time Frame |
|---|---|---|
| Vector Shedding, Measured in Urine, Saliva, and Stool Samples Post-Infusion | Day 1 up to Week 104 | |
| Level of Antibody Titers to Recombinant Adeno-Associated Virus Serotype rh74 (rAAVrh74) | Day 2 up to Week 156 |
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Inclusion Criteria:
All Cohorts:
Exclusion Criteria:
Note: Other inclusion/exclusion criteria apply.
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Sarepta Therapeutics Inc., For Clinical Trial Information, Select Option 4 | Contact | 1-888-SAREPTA (1-888-727-3782) | SareptAlly@Sarepta.com |
| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Sarepta Therapeutics, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Arkansas Children's Hospital | Recruiting | Little Rock | Arkansas | 72202 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 39747998 | Derived | Potter RA, Moeller IH, Khan S, Haegel H, Hollenstein A, Steiner G, Wandel C, Murphy AP, Asher DR, Palatinsky E, Griffin DA, Mason S, Iannaccone ST, Zaidman CM, Rodino-Klapac LR. Immunologic investigations into transgene directed immune-mediated myositis following delandistrogene moxeparvovec gene therapy. Sci Rep. 2025 Jan 2;15(1):4. doi: 10.1038/s41598-024-84077-w. | |
| 37539981 |
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| Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and Adverse Events of Special Interest (AESIs) | Baseline up to Week 156 |
| Cohort 8: Number of Participants With Infections, Edema, Wound-healing Complications, Hyperlipidemia, Angioedema, and Intestinal Lung Disease/ Non-infectious Pneumonitis | Baseline up to Week 72 |
| Change from Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12, as Measured by Immunofluorescence (IF) Fiber Intensity | Baseline, Week 12 |
| Change from Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12, as Measured by IF Percent Dystrophin Positive Fibers (PDPF) | Baseline, Week 12 |
| Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12 as Measured by IF Fiber Intensity | Week 12 |
| Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12 as Measured by IF PDPF | Week 12 |
| Cohort 8: Number of Participants With Hepatic AEs, Hepatic Biomarkers, and Laboratory Assessments Indicative of Either Acute Hepatocellular Injury or Acute Liver Dysfunction | Baseline up to Week 72 |
| Cohort 8: Number of Participants with Severe ALI | Baseline up to Week 72 |
| Cohort 8: Duration of Steroids Administered | Baseline up to Week 72 |
| Stanford University | Recruiting | Palo Alto | California | 94304 | United States |
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| University of California, Davis | Recruiting | Sacramento | California | 95616 | United States |
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| Washington University in St. Louis | Recruiting | St Louis | Missouri | 63110 | United States |
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| Nationwide Children's Hospital | Active, not recruiting | Columbus | Ohio | 43205 | United States |
| Neurology Rare Disease Center | Recruiting | Flower Mound | Texas | 75028 | United States |
|
| Children's Hospital of The King's Daughters | Recruiting | Norfolk | Virginia | 23507 | United States |
|
| Zaidman CM, Proud CM, McDonald CM, Lehman KJ, Goedeker NL, Mason S, Murphy AP, Guridi M, Wang S, Reid C, Darton E, Wandel C, Lewis S, Malhotra J, Griffin DA, Potter RA, Rodino-Klapac LR, Mendell JR. Delandistrogene Moxeparvovec Gene Therapy in Ambulatory Patients (Aged >/=4 to <8 Years) with Duchenne Muscular Dystrophy: 1-Year Interim Results from Study SRP-9001-103 (ENDEAVOR). Ann Neurol. 2023 Nov;94(5):955-968. doi: 10.1002/ana.26755. Epub 2023 Sep 7. |
| ID | Term |
|---|---|
| D020388 | Muscular Dystrophy, Duchenne |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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